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AR128785A1 - LYOPHILIZED COMPOSITIONS COMPRISING RECOMBINANT ADENO-ASSOCIATED VIRUS (rAAV) PARTICLES - Google Patents

LYOPHILIZED COMPOSITIONS COMPRISING RECOMBINANT ADENO-ASSOCIATED VIRUS (rAAV) PARTICLES

Info

Publication number
AR128785A1
AR128785A1 ARP230100629A ARP230100629A AR128785A1 AR 128785 A1 AR128785 A1 AR 128785A1 AR P230100629 A ARP230100629 A AR P230100629A AR P230100629 A ARP230100629 A AR P230100629A AR 128785 A1 AR128785 A1 AR 128785A1
Authority
AR
Argentina
Prior art keywords
raav
particles
concentration
associated virus
recombinant adeno
Prior art date
Application number
ARP230100629A
Other languages
Spanish (es)
Inventor
Dingjiang Liu
Li Zhi
Yao Chen
Kuan Lai
- Wert Jonathan Yu
Xiaoyan Wang
Original Assignee
Regeneron Pharma
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Regeneron Pharma filed Critical Regeneron Pharma
Publication of AR128785A1 publication Critical patent/AR128785A1/en

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/19—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles lyophilised, i.e. freeze-dried, solutions or dispersions
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K35/00—Medicinal preparations containing materials or reaction products thereof with undetermined constitution
    • A61K35/66—Microorganisms or materials therefrom
    • A61K35/76—Viruses; Subviral particles; Bacteriophages
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/16—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite containing nitrogen, e.g. nitro-, nitroso-, azo-compounds, nitriles, cyanates
    • A61K47/18—Amines; Amides; Ureas; Quaternary ammonium compounds; Amino acids; Oligopeptides having up to five amino acids
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/06—Organic compounds, e.g. natural or synthetic hydrocarbons, polyolefins, mineral oil, petrolatum or ozokerite
    • A61K47/26—Carbohydrates, e.g. sugar alcohols, amino sugars, nucleic acids, mono-, di- or oligo-saccharides; Derivatives thereof, e.g. polysorbates, sorbitan fatty acid esters or glycyrrhizin
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K47/00—Medicinal preparations characterised by the non-active ingredients used, e.g. carriers or inert additives; Targeting or modifying agents chemically bound to the active ingredient
    • A61K47/30—Macromolecular organic or inorganic compounds, e.g. inorganic polyphosphates
    • A61K47/34—Macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyesters, polyamino acids, polysiloxanes, polyphosphazines, copolymers of polyalkylene glycol or poloxamers
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K48/00—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy
    • A61K48/0008—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition
    • A61K48/0025—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid
    • A61K48/0041—Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy characterised by an aspect of the 'non-active' part of the composition delivered, e.g. wherein such 'non-active' part is not delivered simultaneously with the 'active' part of the composition wherein the non-active part clearly interacts with the delivered nucleic acid the non-active part being polymeric
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/0012—Galenical forms characterised by the site of application
    • A61K9/0019—Injectable compositions; Intramuscular, intravenous, arterial, subcutaneous administration; Compositions to be administered through the skin in an invasive manner
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605—Excipients; Inactive ingredients
    • A61K9/1617—Organic compounds, e.g. phospholipids, fats
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605—Excipients; Inactive ingredients
    • A61K9/1617—Organic compounds, e.g. phospholipids, fats
    • A61K9/1623—Sugars or sugar alcohols, e.g. lactose; Derivatives thereof; Homeopathic globules
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K9/00—Medicinal preparations characterised by special physical form
    • A61K9/14—Particulate form, e.g. powders, Processes for size reducing of pure drugs or the resulting products, Pure drug nanoparticles
    • A61K9/16—Agglomerates; Granulates; Microbeadlets ; Microspheres; Pellets; Solid products obtained by spray drying, spray freeze drying, spray congealing,(multiple) emulsion solvent evaporation or extraction
    • A61K9/1605—Excipients; Inactive ingredients
    • A61K9/1629—Organic macromolecular compounds
    • A61K9/1641—Organic macromolecular compounds obtained otherwise than by reactions only involving carbon-to-carbon unsaturated bonds, e.g. polyethylene glycol, poloxamers

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Epidemiology (AREA)
  • Animal Behavior & Ethology (AREA)
  • General Health & Medical Sciences (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Molecular Biology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • General Chemical & Material Sciences (AREA)
  • Oil, Petroleum & Natural Gas (AREA)
  • Virology (AREA)
  • Biotechnology (AREA)
  • Genetics & Genomics (AREA)
  • Microbiology (AREA)
  • Mycology (AREA)
  • Inorganic Chemistry (AREA)
  • Dermatology (AREA)
  • Medicinal Preparation (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)

Abstract

Reivindicación 1: Una formulación farmacéutica que comprende (i) partículas de virus adenoasociado recombinante (rAAV), (ii) un agente regulador a una concentración de alrededor de 1 mM a alrededor de 20 mM, (iii) una primera sal a una concentración de alrededor de 10 mM a alrededor de 50 mM, (iv) una segunda sal a una concentración de alrededor de 60 mM a 100 mM, (v) un azúcar a una concentración de alrededor de 1% p/v a alrededor de 10% p/v, (vi) un agente de carga a una concentración de alrededor de 1% p/v a alrededor de 6% p/v, y (vii) un surfactante a una concentración de alrededor de 0,001% p/v a alrededor de 0,1% p/v, en el que la formulación tiene un pH de alrededor de 6,8 a alrededor de 7,8.Claim 1: A pharmaceutical formulation comprising (i) recombinant adeno-associated virus (rAAV) particles, (ii) a regulatory agent at a concentration of about 1 mM to about 20 mM, (iii) a first salt at a concentration of about 10 mM to about 50 mM, (iv) a second salt at a concentration of about 60 mM to 100 mM, (v) a sugar at a concentration of about 1% w/v to about 10% w/ v, (vi) a bulking agent at a concentration of about 1% w/v to about 6% w/v, and (vii) a surfactant at a concentration of about 0.001% w/v to about 0.1 % w/v, wherein the formulation has a pH of about 6.8 to about 7.8.

ARP230100629A 2022-03-14 2023-03-14 LYOPHILIZED COMPOSITIONS COMPRISING RECOMBINANT ADENO-ASSOCIATED VIRUS (rAAV) PARTICLES AR128785A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US202263319657P 2022-03-14 2022-03-14

Publications (1)

Publication Number Publication Date
AR128785A1 true AR128785A1 (en) 2024-06-12

Family

ID=85979703

Family Applications (1)

Application Number Title Priority Date Filing Date
ARP230100629A AR128785A1 (en) 2022-03-14 2023-03-14 LYOPHILIZED COMPOSITIONS COMPRISING RECOMBINANT ADENO-ASSOCIATED VIRUS (rAAV) PARTICLES

Country Status (11)

Country Link
US (1) US20230293724A1 (en)
EP (1) EP4493162A1 (en)
JP (1) JP2025509566A (en)
KR (1) KR20250002229A (en)
CN (1) CN119072302A (en)
AR (1) AR128785A1 (en)
AU (1) AU2023233503A1 (en)
CA (1) CA3245651A1 (en)
IL (1) IL315322A (en)
TW (1) TW202406560A (en)
WO (1) WO2023177626A1 (en)

Families Citing this family (4)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2025076729A1 (en) * 2023-10-11 2025-04-17 朗信启昇(苏州)生物制药有限公司 Pharmaceutical composition comprising aav and preparation method therefor
WO2025114524A1 (en) * 2023-11-30 2025-06-05 Uniqure Biopharma B.V. Formulations for viral drug products
WO2025185606A1 (en) * 2024-03-04 2025-09-12 Nanjing Legend Biotech Co., Ltd. Composition for cryoprotection of viral vector or vlp
WO2025188316A1 (en) * 2024-03-08 2025-09-12 Jaguar Gene Therapy, Llc Aav pharmaceutical formulations and methods thereof

Family Cites Families (5)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
JP2022513034A (en) * 2018-11-14 2022-02-07 レジェンクスバイオ インコーポレーテッド Gene therapy for neuronal ceroid lipofuscinosis
JP2022522995A (en) * 2019-01-18 2022-04-21 ボイジャー セラピューティクス インコーポレイテッド Methods and systems for producing AAV particles
US20220143115A1 (en) * 2019-04-19 2022-05-12 Regenxbio Inc. Adeno-Associated Virus Vector Formulations and Methods
AU2020362119A1 (en) * 2019-10-07 2022-05-26 Regenxbio Inc. Adeno-associated virus vector pharmaceutical composition and methods
TW202144575A (en) * 2020-04-03 2021-12-01 美商拜奧馬林製藥公司 Treatment of phenylketonuria with aav and therapeutic formulations

Also Published As

Publication number Publication date
EP4493162A1 (en) 2025-01-22
CA3245651A1 (en) 2023-09-21
KR20250002229A (en) 2025-01-07
WO2023177626A1 (en) 2023-09-21
IL315322A (en) 2024-10-01
JP2025509566A (en) 2025-04-11
US20230293724A1 (en) 2023-09-21
CN119072302A (en) 2024-12-03
TW202406560A (en) 2024-02-16
AU2023233503A1 (en) 2024-09-19

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